Paediatric research sets new standards for therapy in paediatric and adult cholestasis

Rebecca Jeyaraj, Eamonn R. Maher, Deirdre Kelly

Research output: Contribution to journalReview articlepeer-review

Abstract

Children with Alagille syndrome and progressive familial intrahepatic cholestasis (PFIC) experience debilitating pruritus, for which there have been few effective treatment options. In the past 2 years, the ileal bile acid transporter (IBAT) inhibitors maralixibat and odevixibat have been approved for the management of cholestatic pruritus in these individuals, representing an important step forward in improving their quality of life. Emerging data suggest these drugs might also improve event-free survival, therefore potentially altering the typical disease course currently seen in these disorders. This Review will discuss how genetic advances have clarified the molecular basis of cholestatic disorders, facilitating the development of new therapeutic options that have only been evaluated in children. We focus specifically on the newly licensed IBAT inhibitors for patients with Alagille syndrome and PFIC and explore the next steps for these drugs in relation to other paediatric and adult cholestatic disorders, recognising that they have the potential to benefit a wider group of patients with gastrointestinal and liver disease.

Original languageEnglish
Pages (from-to)75-84
Number of pages10
JournalThe Lancet. Child & adolescent health
Volume8
Issue number1
Early online date22 Nov 2023
DOIs
Publication statusPublished - Jan 2024

Bibliographical note

Copyright © 2023, Elsevier Ltd. This accepted manuscript version is licensed under the Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International License [https://creativecommons.org/licenses/by-nc-nd/4.0/].

Keywords

  • Child
  • Humans
  • Alagille Syndrome/drug therapy
  • Quality of Life
  • Cholestasis/drug therapy
  • Pruritus/drug therapy

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